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botulism - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

botulism

MONDO:0005498

Also known as:botulism poisoninginfection due to Clostridium botulinumfood poisoning due to Clostridium botulinum

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Overview

A serious bacterial infection caused by botulinum toxin which is produced by Clostridium botulinum. Patients are infected usually by ingestion of contaminated food or wound contamination. It leads to muscle paralysis which may result in respiratory failure.

TreatmentsNo approved treatments · 2 in pipeline

Clinical Trials2 registered

Diagnosis

Biomarker and diagnostic research for botulism has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for botulism. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for botulism, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for botulism. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: <1 in 1,000,000 (VERY_RARE).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

2 clinical trials registered. Interventions under study include drug therapy, other interventions, and biologic therapy. Pipeline includes 1 PHASE4, 1 PHASE1. Research is sponsored by a mix of industry and academic institutions.

255 publications have been identified in PubMed for botulism. Research spans Case Report / Case Series (30%), Epidemiology / Natural History (24%), and Basic Science / Preclinical (16%).

Research Type	Count	% of Total
Patient case studies	77

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 11:52 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:1267

GARD

Genetic and Rare Diseases Info Center

GARD:943

Questions for Your Care Team

→Are any of the 2 active clinical trials a good fit for my situation?
→What is the current development status of recombinant botulinum vaccine A/B, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Kisho

botulism

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Botulism Type F, a Rare Type of Botulism that Can Masquerade as a Posterior Fossa Stroke.

Wound Botulism Complicating a Snake Bite Wound Following Traditional Application of Raw Goat Skin Successfully Treated with Botulinum Antitoxin: A Case Report.

People at Increased Risk for Botulism

Botulism Prevention

About Botulism

botulism

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Botulism Type F, a Rare Type of Botulism that Can Masquerade as a Posterior Fossa Stroke.

Wound Botulism Complicating a Snake Bite Wound Following Traditional Application of Raw Goat Skin Successfully Treated with Botulinum Antitoxin: A Case Report.

People at Increased Risk for Botulism

Botulism Prevention

About Botulism